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Natural history of liver disease in a large international cohort of children with Alagille syndrome: Results from the GALA study

by Vandriel, Shannon M, Li, Li-Ting, She, Huiyu, Wang, Jian-She, Gilbert, Melissa A, Jankowska, Irena, Czubkowski, Piotr, Gliwicz-Miedzińska, Dorota, Gonzales, Emmanuel M, Jacquemin, Emmanuel, Bouligand, Jérôme, Spinner, Nancy B, Loomes, Kathleen M, Piccoli, David A, D'Antiga, Lorenzo, Nicastro, Emanuele, Sokal, Étienne, Demaret, Tanguy, Ebel, Noelle H, Feinstein, Jeffrey A, Fawaz, Rima, Nastasio, Silvia, Lacaille, Florence, Debray, Dominique, Arnell, Henrik, Fischler, Björn, Siew, Susan, Stormon, Michael, Karpen, Saul J, Romero, Rene, Kim, Kyung Mo, Baek, Woo Yim, Hardikar, Winita, Shankar, Sahana, Roberts, Amin J, Evans, Helen M, Jensen, M Kyle, Kavan, Marianne, Sundaram, Shikha S, Chaidez, Alexander, Karthikeyan, Palaniswamy, Sanchez, Maria Camila, Cavalieri, Maria Lorena, Verkade, Henkjan J, Lee, Way Seah, Squires, James E, Hajinicolaou, Christina, Lertudomphonwanit, Chatmanee, Fischer, Ryan T, Larson-Nath, Catherine, Mozer-Glassberg, Yael, Arikan, Cigdem, Lin, Henry C, Bernabeu, Jesus Quintero, Alam, Seema, Kelly, Deirdre A, Carvalho, Elisa, Ferreira, Cristina Targa, Indolfi, Giuseppe, Quiros-Tejeira, Ruben E, Bulut, Pinar, Calvo, Pier Luigi, Önal, Zerrin, Valentino, Pamela L, Desai, Dev M, Eshun, John, Rogalidou, Maria, Dezsőfi, Antal, Wiecek, Sabina, Nebbia, Gabriella, Pinto, Raquel Borges, Wolters, Victorien M, Tamara, María Legarda, Zizzo, Andréanne N, Garcia, Jennifer, Schwarz, Kathleen, Beretta, Marisa, Sandahl, Thomas Damgaard, Jimenez-Rivera, Carolina, Kerkar, Nanda, Brecelj, Jernej, Mujawar, Quais, Rock, Nathalie, Busoms, Cristina Molera, Karnsakul, Wikrom, Lurz, Eberhard, Santos-Silva, Ermelinda, Blondet, Niviann, Bujanda, Luis, Shah, Uzma, Thompson, Richard J, Hansen, Bettina E, Kamath, Binita M
Published in Hepatology (Baltimore, Md.) (01.02.2023)

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Event-free survival of maralixibat-treated patients with Alagille syndrome compared to a real-world cohort from GALA

by Hansen, Bettina E, Vandriel, Shannon M, Vig, Pamela, Garner, Will, Mogul, Douglas B, Loomes, Kathleen M, Piccoli, David A, Rand, Elizabeth B, Jankowska, Irena, Czubkowski, Piotr, Gliwicz-Miedzińska, Dorota, Gonzales, Emmanuel M, Jacquemin, Emmanuel, Bouligand, Jérôme, D'Antiga, Lorenzo, Nicastro, Emanuele, Arnell, Henrik, Fischler, Björn, Sokal, Étienne, Demaret, Tanguy, Siew, Susan, Stormon, Michael, Karpen, Saul J, Romero, Rene, Ebel, Noelle H, Feinstein, Jeffrey A, Roberts, Amin J, Evans, Helen M, Sundaram, Shikha S, Chaidez, Alexander, Hardikar, Winita, Shankar, Sahana, Fischer, Ryan T, Lacaille, Florence, Debray, Dominique, Lin, Henry C, Jensen, M Kyle, Jaramillo, Catalina, Karthikeyan, Palaniswamy, Indolfi, Giuseppe, Verkade, Henkjan J, Larson-Nath, Catherine, Quiros-Tejeira, Ruben E, Valentino, Pamela L, Rogalidou, Maria, Dezsőfi, Antal, Squires, James E, Schwarz, Kathleen, Calvo, Pier Luigi, Bernabeu, Jesus Quintero, Zizzo, Andréanne N, Nebbia, Gabriella, Bulut, Pinar, Santos-Silva, Ermelinda, Fawaz, Rima, Nastasio, Silvia, Karnsakul, Wikrom, Tamara, María Legarda, Busoms, Cristina Molera, Kelly, Deirdre A, Sandahl, Thomas Damgaard, Jimenez-Rivera, Carolina, Banales, Jesus M, Mujawar, Quais, Li, Li-Ting, She, Huiyu, Wang, Jian-She, Kim, Kyung Mo, Oh, Seak Hee, Sanchez, Maria Camila, Cavalieri, Maria Lorena, Lee, Way Seah, Hajinicolaou, Christina, Lertudomphonwanit, Chatmanee, Waisbourd-Zinman, Orith, Arikan, Cigdem, Alam, Seema, Carvalho, Elisa, Melere, Melina, Eshun, John, Önal, Zerrin, Desai, Dev M, Wiecek, Sabina, Pinto, Raquel Borges, Wolters, Victorien M, Garcia, Jennifer, Beretta, Marisa, Kerkar, Nanda, Brecelj, Jernej, Rock, Nathalie, Lurz, Eberhard, Blondet, Niviann, Shah, Uzma, Thompson, Richard J, Kamath, Binita M
Published in Hepatology (Baltimore, Md.) (01.06.2024)

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OC7 Maralixibat-treated patients with Alagille syndrome demonstrate event-free survival in a natural history comparison with patients with from the GALA database: application of real-world evidence analytics

by Hansen, B E, Vandriel, S M, Vig, P, Garner, W, Mogul, D B, Loomes, K M, Piccoli, DA, Rand, E B, Jankowska, I, Czubkowski, P, Gliwicz-Miedzińska, D, Gonzales, E M, Jacquemin, E, Bouligand, J, L D’Antiga, Nicastro, E, Arnell, H, Fischler, B, Sokal, É, Demaret, T, Siew, S, Stormon, M, Karpen, S J, Romero, R, Ebel, N H, Feinstein, JA, Roberts, A J, Evans, H M, Sundaram, S S, Chaidez, A, Hardikar, W, Shankar, S, Fischer, R T, Lacaille, F, Debray, D, Lin, H C, Jensen, M K, Jaramillo, C, Karthikeyan, P, Indolfi, G, Verkade, HJ, Larson-Nath, C, Quiros-Tejeira, R E, Valentino, P L, Rogalidou, M, Dezsőfi, A, Squires, JE, Schwarz, K, Calvo, P L, J Quintero Bernabeu, Zizzo, AN, Nebbia, G, Bulut, P, Santos-Silva, E, Fawaz, R, Nastasio, S, Karnsakul, W, M Legarda Tamara, C Molera Busoms, Kelly, D, T Damgaard Sandahl, Jimenez-Rivera, C, Banales, JM, Mujawar, Q, Li, L T, She, H, Wang, J S, Kim, K M, Oh, SH, Sanchez, M C, Cavalieri, M L, Lee, W S, Hajinicolaou, C, Lertudomphonwanit, C, Waisbourd-Zinman, O, Arikan, C, Alam, S, Carvalho, E, Melere, M, Eshun, J, Önal, Z, Desai, D M, Wiecek, S, Pinto, R B, Wolters, V M, Garcia, J, Beretta, M, Kerkar, N, Brecelj, J, Rock, N, Lurz, E, Blondet, N, Shah, U, Thompson, R J, Kamath, B M
Published in Frontline gastroenterology (01.07.2023)

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